Novartis has secured US Food and Drug Administration (FDA) accelerated approval for Fabhalta (iptacopan) for reducing proteinuria in adults with primary IgA nephropathy (IgAN), a rare, progressive kidney disease.
Fabhalta is an alternative complement pathway inhibitor.
The approval is based on pre-specified interim results from the Phase III APPLAUSE-IgAN study, which showed a 44% reduction in proteinuria at nine months compared to placebo.
The APPLAUSE-IgAN study is a multicentre, randomised, double-blind, placebo-controlled trial involving 518 adult IgAN patients.
It assesses the safety and efficacy of Fabhalta, with proteinuria reduction and estimated glomerular filtration rate decline over 24 months as primary endpoints.
Secondary endpoints include urine protein-to-creatinine ratio reduction, composite kidney failure events and changes in fatigue levels.
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Continued approval depends on further evidence of clinical benefit, with final study results expected in 2025.
The APPLAUSE-IgAN study’s interim analysis is focused on proteinuria reduction, a key marker of kidney damage. Fabhalta demonstrated a significant 38% reduction in proteinuria compared to placebo.
It included patients on stable doses of renin-angiotensin system inhibitors, with or without sodium-glucose cotransporter-2, and Fabhalta’s safety profile was consistent with previous findings.
Novartis is also developing two additional IgAN therapies, atrasentan and zigakibart, in late-stage trials.
Novartis US president Victor Bultó stated: “Today’s approval of Fabhalta as a first-in-class medicine for IgA nephropathy is an important milestone in our journey to evolve rare renal disease care by bringing new treatments to people in urgent need of options.
“We are deeply committed to those living with rare renal diseases and look forward to continued partnership with this community as we further advance our broad portfolio.”
Atrasentan has received FDA filing acceptance, and zigakibart is currently in Phase III development.
